From left to right: Dr. Gerald Lipshutz, Dr. Zulema Romero Garcia and Dr. Donald Kohn Researchers at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA have received more than $16 million in grants from the California Institute for Regenerative Medicine, the state’s stem cell agency, to [...]

By Heather McKenzie See Orignial Story Here Arginase 1 deficiency, or ARG1-D, is a rare, inherited disorder occurring in approximately 1.1 in 1 million live U.S. births. While born with normal function, by the age of two, patients with the condition have progressive deterioration in their physical ability due to the gradual accumulation of arginine and ammonia [...]

Stockholm, October 13, 2023: Immedica today announces that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion recommending marketing authorization of Loargys® (pegzilarginase) for the treatment of arginase 1 deficiency (ARG1-D) in patients two years and older. The positive opinion from the CHMP is now referred [...]

The FDA wouldnʼt review a drug that was 90% effective. In rare diseases, this is too common, said Ultragenyx CEO Dr. Emil Kakkis and a leading rare disease researcher.

Aeglea BioTherapeutics Announces Sale of Pegzilarginase to Immedica Pharma Global rights to pegzilarginase in development for Arginase 1 Deficiency sold to Immedica Pharma for $15 million upfront cash proceeds and up to $100 million of contingent milestone payments Marketing Authorisation Application for pegzilarginase is under review by the European Medicines Agency WALTHAM, Mass., July 27, [...]

Arginase 1 Deficiency Foundation Files Petition Urging Aeglea Biotherapeutics to Reinstate Life-Saving Drug Following Abrupt Withdrawal to Clinical Trial Patients in the United States Seattle, Washington, April 21, 2023: The Arginase 1 Deficiency Foundation, a leading advocacy group for individuals and families impacted by the debilitating rare metabolic disease, has launched a petition demanding that [...]

Pegzilarginase Clinical Trial Update (January 2023) As Aeglea previously announced, we are in the process of concluding our ongoing pegzilarginase Phase 1 and Phase 3 clinical trials and transitioning those patients to a new study. All patients in our two pegzilarginase clinical trials received their last dose before the end of December, and final [...]

Aeglea BioTherapeutics Submits BLA to FDA for Pegzilarginase for the Treatment of Arginase 1 Deficiency SEE ORIGINAL STORY HERE BLA submission provides FDA with all pegzilarginase program data to review in detail; Aeglea looks forward to working collaboratively with the FDA on next steps in the review process If approved, pegzilarginase would be the [...]

REDACTED - FDA Family-Led Listening Session Report On June 10, 2021, a group of patients and families affected by Arginase 1 Deficiency (ARG1-D) participated in a one-hour virtual listening session with officials from the Food and Drug Administration (FDA). The session enabled those with ARG1-D and their caregivers to  share their experiences, perspectives, needs, goals [...]

We’re proud to partner with the Child Neurology Foundation to conduct a survey to understand caregivers’ experiences on their journey in child neurology. Results will be shared with advocates and health care providers, so they are better able serve families. Caregivers of children with neurologic conditions can complete the brief survey thru April 1st at: www.surveymonkey.com/r/2022CNFCaregiverAssessment